JAMA Neurology has published a paper titled, “Safety and Clinical Effects of Mesenchymal Stem Cells Secreting Neurotrophic Factor Transplantation in Patients With Amyotrophic Lateral Sclerosis – Results of Phase1/2 and 2a Clinical Trials.” This study comes out of a collaboration of researchers from BrainStorm Cell Therapeutics and the Hadassah-Hebrew University Medical Center both located in Israel.
The researchers set out to understand the safety and clinical effects of treatment with mesenchymal stem cells induced to secrete neurotrophic factors (MSC-NTF) in patients with ALS. Stem cells used in this study are adult mesenchymal stem cells derived from bone marrow samples given by participants in the trial. The bone marrow cells are turned into stem cells that secrete the NTFs. NTFs are a type of nutrient for cells and were previously shown to have protective effects in animal models of neurodegenerative disease.
The new study showed that implanting stem cells that produce neurotrophic factors (MSC-NTF) into either muscle or the intrathecal space surrounding the spinal cord, or both, after 6 months were shown to be safe and associated with only mild and transient adverse effects, principally headache and fever. The study is the first in which stem cells that have been modified to secrete neurotrophic factors have been implanted in people with ALS.
The study included 26 patients in all. Measurements of breathing ability and overall function suggested the treatment may have been associated with small reductions in the rate of disease progression. These results must be treated with caution, since the trial did not include a placebo comparator, making it difficult to know whether the effects were due to the treatment or to some other factor.
“We are encouraged to see that implantation of modified stem cells in this trial was safe for people with ALS,” said Association Chief Scientist Lucie Bruijn, Ph.D., M.B.A. “We look forward to learning more about the safety and efficacy of this therapeutic approach, as more trials are performed and results are reported.”
Information on additional clinical trials, including those exploring the potential of stem cell implantation, can be found on the NEALS website.